Press Release: Pivotal data demonstrate once-weekly efanesoctocog
alfa provides superior bleed protection compared to prior factor
prophylaxis
Pivotal data demonstrate once-weekly efanesoctocog alfa provides
superior bleed protection compared to prior factor prophylaxis
- Investigational
once-weekly efanesoctocog alfa prophylaxis met the primary efficacy
endpoint providing clinically meaningful bleed protection for
people with severe hemophilia A
- Results underscore
the ability of efanesoctocog alfa to sustain normal to near-normal
factor levels and the potential to transform prophylactic
treatment, providing people with hemophilia A with higher
protection for longer
- Additional data
showed efanesoctocog alfa prophylaxis resulted in statistically
significant and clinically meaningful improvements in physical
health, pain intensity and joint health in patients on prior factor
VIII prophylaxis
Paris and Stockholm – July
10, 2022 – Sanofi and Swedish
Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) presented for the
first time today, in a late-breaking session at the 30th
International Society on Thrombosis and Haemostasis (ISTH)
Congress, positive results from the XTEND-1 pivotal Phase 3 study
evaluating the safety, efficacy and pharmacokinetics of
efanesoctocog alfa (BIVV001), an investigational factor VIII
replacement therapy, in previously treated adults and adolescents
≥12 years with severe hemophilia A.
The study met the primary efficacy endpoint,
with once-weekly efanesoctocog alfa prophylaxis providing
clinically meaningful bleed protection for people with severe
hemophilia A. The median and mean annualized bleeding rates (ABR)
were 0.00 (IQR: 0.00-1.04) and 0.71 (SD: 1.43) respectively. The
study also met the key secondary endpoint, demonstrating superior
bleed protection (p<0.0001) over prior factor VIII prophylaxis
with an estimated ABR reduction of 77% and a mean ABR of 0.69
compared to 2.96 on prior prophylaxis, based on an intra-patient
comparison (n=78). In a subset of participants (n=17) studied at
baseline and week 26, mean factor VIII levels remained in the
normal to near-normal range (>40 IU/dL) for the majority of the
week, and at 15 IU/dL at Day seven post-dose, providing increased
factor activity level protection for patients with once-weekly
prophylaxis.
Annette von Drygalski, MD,
PharmDInvestigator, Professor and Director, Hemophilia and
Thrombosis Treatment Center, UC San Diego“The phase 3 data
demonstrate once-weekly efanesoctocog alfa’s potential to provide
superior bleed protection, leading to substantial improvements in
physical health, pain and joint health, by sustaining high factor
levels for the majority of the week. These unprecedented results
may offer people with hemophilia A the possibility to redefine
their treatment expectations.”
Data show adults and adolescents treated with
once-weekly efanesoctocog alfa experienced statistically
significant and clinically meaningful improvements in physical
health (p=0.0001), pain intensity (p=0.0276), and joint health
(p=0.0101) when comparing week 52 and baseline measurements.i
Moreover, efanesoctocog alfa was effective at treating bleeds,
including in target joints; 96.7% of bleeds were resolved with a
single 50 IU/kg dose. Efanesoctocog alfa was well tolerated and
inhibitor development to factor VIII was not detected. The most
common treatment-emergent adverse events (>5% of participants
overall) were headache, arthralgia, fall, and back pain.
Dietmar Berger, MD, PhDGlobal
Head of Development and Chief Medical Officer, Sanofi “We are
committed to advancing innovative medicines that disrupt the
status-quo and address the unmet needs that persist for people with
rare conditions like hemophilia. These robust data illustrate the
promise of efanesoctocog alfa’s efficacy with once-weekly dosing
and underscore its potential as a therapy with best-in-disease
efficacy.”
Anders Ullman, MD,
PhDHead of R&D and Chief Medical Officer, Sobi “We
believe transforming the treatment paradigm for hemophilia A can
only be achieved through elevating standards of care towards normal
hemostasis. These data demonstrate the profile of efanesoctocog
alfa in significant clinical terms, and further strengthen its
potential to ultimately improve the lives of many living with this
condition.”
The U.S Food and Drug Administration (FDA)
granted efanesoctocog alfa Breakthrough Therapy Designation in May
2022, Fast Track designation in February 2021 and Orphan Drug
designation in August 2017. The European Commission also granted
efanesoctocog alfa Orphan Drug designation in June 2019. Regulatory
submission of the Biologics License Application to the U.S. FDA
occurred in June 2022 and submission in the EU will follow
availability of data from the ongoing XTEND-Kids pediatric study,
expected in 2023.
About Phase 3 XTEND-1 Study (NCT04161495)The
Phase 3 XTEND-1 study (NCT04161495) was an open-label,
non-randomized interventional study assessing the safety, efficacy
and pharmacokinetics of once-weekly efanesoctocog alfa in people 12
years of age or older (n=159) with severe hemophilia A who were
previously treated with factor VIII replacement therapy. The study
consists of two parallel treatments arms — the prophylaxis Arm A
(n=133), in which patients who had received prior factor VIII
prophylaxis began receiving once-weekly intravenous efanesoctocog
alfa prophylaxis (50 IU/kg) for 52 weeks, and the on-demand Arm B
(n=26), in which patients who had received prior on-demand factor
VIII therapy began 26 weeks of on-demand efanesoctocog alfa (50
IU/kg), then switched to once-weekly prophylaxis (50 IU/kg) for an
additional 26 weeks.
The primary efficacy endpoint was the ABR in Arm
A, and the key secondary endpoint was an intra-patient comparison
of ABR during the efanesoctocog alfa weekly prophylaxis treatment
period versus the prior factor VIII prophylaxis ABR for
participants in Arm A who had participated in a previous
observational study (Study 242HA201/OBS16221).
About hemophilia AHemophilia A is a rare,
genetic disorder in which the ability of a person’s blood to clot
is impaired due to a lack of factor VIII. Hemophilia A occurs in
about one in 5,000 male births annually, and more rarely in
females. People with hemophilia can experience bleeding episodes
that can cause pain, irreversible joint damage and life-threatening
hemorrhages. Factor replacement therapy remains a cornerstone of
care and can be used across multiple treatment scenarios.
About efanesoctocog alfaEfanesoctocog alfa,
formerly BIVV001, is a novel and investigational recombinant factor
VIII therapy that is designed to extend protection from bleeds with
once-weekly prophylactic dosing for people with hemophilia A. It
builds on the innovative Fc fusion technology by adding a region of
von Willebrand factor and XTEN® polypeptides to extend its time in
circulation. It is the first investigational factor VIII therapy
that has been shown to break through the von Willebrand factor
ceiling, which imposes a half-life limitation on current factor
VIII therapies. Efanesoctocog alfa is currently under clinical
investigation and its safety and efficacy have not been evaluated
by any regulatory authority.
About the Sanofi and Sobi collaborationSobi and
Sanofi collaborate on the development and commercialization of
Alprolix® and Elocta®/Eloctate®.The companies also collaborate on
the development and commercialization of efanesoctocog alfa, an
investigational factor VIII therapy with the potential to provide
high sustained factor activity levels with once-weekly dosing for
people with hemophilia A. Sobi has final development and
commercialization rights in the Sobi territory (essentially Europe,
North Africa, Russia and most Middle Eastern markets). Sanofi has
final development and commercialization rights in North America and
all other regions in the world excluding the Sobi territory.
About Sobi®Sobi is a specialised international
biopharmaceutical company transforming the lives of people with
rare diseases. Providing sustainable access to innovative medicines
in the areas of haematology, immunology and specialty care, Sobi
has approximately 1,600 employees across Europe, North America, the
Middle East and Asia. In 2021, revenue amounted to SEK 15.5
billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm.
More about Sobi at sobi.com, LinkedIn and YouTube.
About SanofiWe are an innovative global
healthcare company, driven by one purpose: we chase the miracles of
science to improve people’s lives. Our team, across some 100
countries, is dedicated to transforming the practice of medicine by
working to turn the impossible into the possible. We provide
potentially life-changing treatment options and life-saving vaccine
protection to millions of people globally, while putting
sustainability and social responsibility at the center of our
ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
Media RelationsSandrine
Guendoul | + 33 6 25 09 14 25
| sandrine.guendoul@sanofi.comSally
Bain | + 1 617 834 6026
| sally.bain@sanofi.comKate
Conway | + 1 508 364
4931 | lisa.zobel@sanofi.com
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| eva.schaefer-jansen@sanofi.comArnaud
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arnaud.delepine@sanofi.comCorentine
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corentine.driancourt@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
felix.lauscher@sanofi.comPriya
Nanduri | +1 617 764 6418 |
priya.nanduri@sanofi.com Nathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
Sobi Contacts:Media RelationsFor Sobi Media
contacts, click here.
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the Sobi Investor Relations Team, click here.
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i Physical health was assessed with the
Haem-A-QoL Physical Health score. Pain intensity was assessed using
the PROMIS Pain Intensity 3a past 7 days intensity of pain at its
worst score. Joint health was assessed using the Hemophilia Joint
Health score.
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